Har­mo­ny Bio­sciences sets terms on $100M IPO; FDA ac­cepts a Pro­tal­ix BLA for re­view

Paragon port­fo­lio com­pa­ny Har­mo­ny Bio­sciences an­nounced its IPO terms on Tues­day, which set a range of $20 and $23 a share.

The Penn­syl­va­nia-based com­pa­ny, which ini­tial­ly filed for a $100 mil­lion IPO weeks ago, plans on of­fer­ing 4.7 mil­lion shares. It seeks to raise rough­ly $100 mil­lion, which will fund the clin­i­cal ad­vance­ment of its drug pitolisant.

Jeff Aronin — who serves as chair­man of the Har­mo­ny board — stirred up con­tro­ver­sy sev­er­al years ago when he di­rect­ed an old steroid he picked up cheap to Duchenne MD pa­tients for $89,000. But that isn’t the on­ly dra­ma on the ex­ec­u­tive board — for­mer CEO Bob Re­pel­la pled guilty to mail fraud in May in a col­lege ad­mis­sions scan­dal.

Har­mo­ny ac­quired pitolisant from Bio­pro­jet in France, and now sells it as Wak­ix to treat ex­ces­sive day­time sleepi­ness for adults with nar­colep­sy. Plans are in place for oth­er in­di­ca­tions, in­clud­ing pe­di­atric EDS and cat­a­plexy, ac­cord­ing to the S-1. It’s ex­pect­ed to en­ter a Phase III clin­i­cal tri­al in pe­di­atric pa­tients lat­er this year, and re­sub­mit a re­sponse to the FDA for an adult cat­a­plexy in­di­ca­tion. — Nicole De­Feud­is

FDA ac­cepts Pro­tal­ix li­cense ap­pli­ca­tion for drug that treats Fab­ry dis­ease

An Is­raeli biotech that de­vel­ops ther­a­pies from plant-based pro­teins got some good news from US reg­u­la­tors Tues­day.

Pro­tal­ix Bio­Ther­a­peu­tics an­nounced that the FDA has ac­cept­ed the com­pa­ny’s bi­o­log­ics li­cense ap­pli­ca­tion for pe­gu­ni­gal­si­dase al­fa (or PRX-102) and grant­ed the can­di­date pri­or­i­ty re­view for the treat­ment of adults with Fab­ry dis­ease. The PDU­FA date set by reg­u­la­tors is Jan­u­ary 27, 2021.

The biotech has been de­vel­op­ing PRX-102, a plant cell cul­ture-ex­pressed and chem­i­cal­ly mod­i­fied ver­sion of the al­pha-Galac­tosi­dase-A en­zyme, with a sub­sidiary of Ital­ian phar­ma Chiesi. In clin­i­cal stud­ies, PRX-102 has been ob­served to have a cir­cu­la­to­ry half-life of ap­prox­i­mate­ly 80 hours.

Fab­ry dis­ease is a rare ge­net­ic dis­or­der that pre­vents the body from mak­ing the al­pha-Galac­tosi­dase-A en­zyme, re­sult­ing in ab­nor­mal de­posits of fat­ty sub­stances in blood ves­sel walls. The ul­ti­mate con­se­quences of these de­posits can range from pain and im­paired pe­riph­er­al sen­sa­tion to kid­ney and heart fail­ure. — Max Gel­man

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

Anthony Coyle (Pfizer via Youtube)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-Suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.